Making gene editing a therapeutic reality.

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Abstract

This review discusses current bottlenecks in making CRISPR-Cas9-mediated genome editing a therapeutic reality and it outlines recent strategies that aim to overcome these hurdles as well as the scope of current clinical trials that pioneer the medical translation of CRISPR-Cas9. Additionally, this review outlines the specifics of disease-modifying gene editing in recessive versus dominant genetic diseases with the focus on genetic myopathies that are exemplified by Duchenne muscular dystrophy and myotonic dystrophies.

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APA

Conboy, I., Murthy, N., Etienne, J., & Robinson, Z. (2018). Making gene editing a therapeutic reality. F1000Research. F1000 Research Ltd. https://doi.org/10.12688/f1000research.16106.1

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